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WASHINGTON: An experimental gene therapy has helped
restore partial vision to persons with congenital retinal disease,
according to studies published Sunday in a breakthrough that
provides hope for treating various eye illnesses.
In one study, clinical trials
showed success on three young adults at Children’s Hospital of
Philadelphia who suffered from a rare and as yet incurable form of
congenital blindness.
The retinal degenerations include
Leber congenital amaurosis, or LCA, a group of diseases that affect
light receptors in the retina beginning in early childhood and often
causing total blindness in patients in their twenties or thirties.
“This result is important for
the entire field of gene therapy,” study leader Katherine High
told the New England Journal of Medicine whose website reported the
findings by a collection of international doctors and scientists.
“Gene transfer has been in
clinical trials for over 15 years now, and although it has an
excellent safety record, examples of therapeutic effect are still
relatively few,” High said.
“The results in this study
provide objective evidence of improvement in the ability to perceive
light, and thus lay the groundwork for future studies in this and
other retinal disorders,” she said.
Scientists used a genetically
engineered virus known as a vector to carry a normal version of the
gene known as RPE65, which is mutated in a form of LCA, to the
patients via surgical procedures performed between October 2007 and
January 2008.
--AFP
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